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134 related items for PubMed ID: 38733639

  • 1. Empagliflozin for treating neutropenia and neutrophil dysfunction in 21 infants with glycogen storage disease 1b.
    Grünert SC, Gautschi M, Baker J, Boyer M, Burlina A, Casswall T, Corpeleijn W, Çıki K, Cotter M, Crushell E, Derks TGJ, Haas D, Kilavuz S, Kingma SDK, Korman SH, Kozek A, de Laet C, Mundy H, Nassogne MC, Quintero V, Rossi A, Spenger J, Spiegel R, Stephenne X, Stojkov D, Tal G, Veiga-da Cunha M, Wortmann SB.
    Mol Genet Metab; 2024 Jun; 142(2):108486. PubMed ID: 38733639
    [Abstract] [Full Text] [Related]

  • 2. Case report: The success of empagliflozin therapy for glycogen storage disease type 1b.
    Klinc A, Groselj U, Mlinaric M, Homan M, Markelj G, Mezek Novak A, Sirca Campa A, Sikonja J, Battelino T, Zerjav Tansek M, Drole Torkar A.
    Front Endocrinol (Lausanne); 2024 Jun; 15():1365700. PubMed ID: 38919482
    [Abstract] [Full Text] [Related]

  • 3. Treating neutropenia and neutrophil dysfunction in glycogen storage disease type Ib with an SGLT2 inhibitor.
    Wortmann SB, Van Hove JLK, Derks TGJ, Chevalier N, Knight V, Koller A, Oussoren E, Mayr JA, van Spronsen FJ, Lagler FB, Gaughan S, Van Schaftingen E, Veiga-da-Cunha M.
    Blood; 2020 Aug 27; 136(9):1033-1043. PubMed ID: 32294159
    [Abstract] [Full Text] [Related]

  • 4. Efficacy and safety of empagliflozin in glycogen storage disease type Ib: Data from an international questionnaire.
    Grünert SC, Derks TGJ, Adrian K, Al-Thihli K, Ballhausen D, Bidiuk J, Bordugo A, Boyer M, Bratkovic D, Brunner-Krainz M, Burlina A, Chakrapani A, Corpeleijn W, Cozens A, Dawson C, Dhamko H, Milosevic MD, Eiroa H, Finezilber Y, Moura de Souza CF, Garcia-Jiménez MC, Gasperini S, Haas D, Häberle J, Halligan R, Fung LH, Hörbe-Blindt A, Horka LM, Huemer M, Uçar SK, Kecman B, Kilavuz S, Kriván G, Lindner M, Lüsebrink N, Makrilakis K, Mei-Kwun Kwok A, Maier EM, Maiorana A, McCandless SE, Mitchell JJ, Mizumoto H, Mundy H, Ochoa C, Pierce K, Fraile PQ, Regier D, Rossi A, Santer R, Schuman HC, Sobieraj P, Spenger J, Spiegel R, Stepien KM, Tal G, Tanšek MZ, Torkar AD, Tchan M, Thyagu S, Schrier Vergano SA, Vucko E, Weinhold N, Zsidegh P, Wortmann SB.
    Genet Med; 2022 Aug 27; 24(8):1781-1788. PubMed ID: 35503103
    [Abstract] [Full Text] [Related]

  • 5. Treatment recommendations for glycogen storage disease type IB- associated neutropenia and neutrophil dysfunction with empagliflozin: Consensus from an international workshop.
    Grünert SC, Derks TGJ, Mundy H, Dalton RN, Donadieu J, Hofbauer P, Jones N, Uçar SK, LaFreniere J, Contreras EL, Pendyal S, Rossi A, Schneider B, Spiegel R, Stepien KM, Wesol-Kucharska D, Veiga-da-Cunha M, Wortmann SB.
    Mol Genet Metab; 2024 Mar 27; 141(3):108144. PubMed ID: 38277989
    [Abstract] [Full Text] [Related]

  • 6. Neutrophil functions in patients with neutropenia due to glycogen storage disease type 1b treated with empagliflozin.
    Kaczor M, Malicki S, Folkert J, Dobosz E, Bryzek D, Chruscicka-Smaga B, Greczan M, Wesół-Kucharska D, Piątosa B, Samborowska E, Madzio J, Książyk J, Ehmke Vel Emczyńska E, Hajdacka M, Potempa J, Młynarski W, Rokicki D, Veillard F.
    Blood Adv; 2024 Jun 11; 8(11):2790-2802. PubMed ID: 38531056
    [Abstract] [Full Text] [Related]

  • 7. Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report.
    Rossi A, Miele E, Fecarotta S, Veiga-da-Cunha M, Martinelli M, Mollica C, D'Armiento M, Mozzillo E, Strisciuglio P, Derks TGJ, Staiano A, Parenti G.
    Ital J Pediatr; 2021 Jul 02; 47(1):149. PubMed ID: 34215305
    [Abstract] [Full Text] [Related]

  • 8. Clinical spectrum, over 12-year follow-up and experience of SGLT2 inhibitors treatment on patients with glycogen storage disease type Ib: a single-center retrospective study.
    Shao YX, Liang CL, Su YY, Lin YT, Lu ZK, Lin RZ, Zhou ZZ, Zeng CH, Tao CY, Liu ZC, Zhang W, Liu L.
    Orphanet J Rare Dis; 2024 Apr 11; 19(1):155. PubMed ID: 38605407
    [Abstract] [Full Text] [Related]

  • 9. [Investigation on the treatment of empagliflozin in glycogen storage disease type Ⅰb].
    Jiang JJ, Ma MS, Wei M, Qiu ZQ.
    Zhonghua Er Ke Za Zhi; 2024 Jun 02; 62(6):526-529. PubMed ID: 38763873
    [Abstract] [Full Text] [Related]

  • 10. Improved inflammatory bowel disease, wound healing and normal oxidative burst under treatment with empagliflozin in glycogen storage disease type Ib.
    Grünert SC, Elling R, Maag B, Wortmann SB, Derks TGJ, Hannibal L, Schumann A, Rosenbaum-Fabian S, Spiekerkoetter U.
    Orphanet J Rare Dis; 2020 Aug 24; 15(1):218. PubMed ID: 32838757
    [Abstract] [Full Text] [Related]

  • 11. Assessment of the diagnosis, treatment, and follow-up of a group of Turkish pediatric glycogen storage disease type 1b patients with varying clinical presentations and a novel mutation.
    Küçükçongar Yavaş A, Engin Erdal A, Bilginer Gürbüz B, Ünlüsoy Aksu A, Kasapkara ÇS.
    J Pediatr Endocrinol Metab; 2023 Nov 27; 36(11):1092-1099. PubMed ID: 37791580
    [Abstract] [Full Text] [Related]

  • 12. Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.
    Calderwood S, Kilpatrick L, Douglas SD, Freedman M, Smith-Whitley K, Rolland M, Kurtzberg J.
    Blood; 2001 Jan 15; 97(2):376-82. PubMed ID: 11154211
    [Abstract] [Full Text] [Related]

  • 13. Understanding the role of SGLT2 inhibitors in glycogen storage disease type Ib: the experience of one UK centre.
    Halligan RK, Dalton RN, Turner C, Lewis KA, Mundy HR.
    Orphanet J Rare Dis; 2022 May 12; 17(1):195. PubMed ID: 35549996
    [Abstract] [Full Text] [Related]

  • 14. Granulocyte colony-stimulating factor in glycogen storage disease type 1b. Results of the European Study on Glycogen Storage Disease Type 1.
    Visser G, Rake JP, Labrune P, Leonard JV, Moses S, Ullrich K, Wendel U, Groenier KH, Smit GP.
    Eur J Pediatr; 2002 Oct 12; 161 Suppl 1():S83-7. PubMed ID: 12373578
    [Abstract] [Full Text] [Related]

  • 15. In vitro and in vivo effects of granulocyte colony-stimulating factor on neutrophils in glycogen storage disease type 1B: granulocyte colony-stimulating factor therapy corrects the neutropenia and the defects in respiratory burst activity and Ca2+ mobilization.
    McCawley LJ, Korchak HM, Douglas SD, Campbell DE, Thornton PS, Stanley CA, Baker L, Kilpatrick L.
    Pediatr Res; 1994 Jan 12; 35(1):84-90. PubMed ID: 7510873
    [Abstract] [Full Text] [Related]

  • 16. Successful use of empagliflozin to treat neutropenia in two G6PC3-deficient children: Impact of a mutation in SGLT5.
    Boulanger C, Stephenne X, Diederich J, Mounkoro P, Chevalier N, Ferster A, Van Schaftingen E, Veiga-da-Cunha M.
    J Inherit Metab Dis; 2022 Jul 12; 45(4):759-768. PubMed ID: 35506446
    [Abstract] [Full Text] [Related]

  • 17. Neutropenia in glycogen storage disease Ib: outcomes for patients treated with granulocyte colony-stimulating factor.
    Dale DC, Bolyard AA, Marrero T, Kelley ML, Makaryan V, Tran E, Leung J, Boxer LA, Kishnani PS, Austin S, Wanner C, Ferrecchia IA, Khalaf D, Maze D, Kurtzberg J, Zeidler C, Welte K, Weinstein DA.
    Curr Opin Hematol; 2019 Jan 12; 26(1):16-21. PubMed ID: 30451720
    [Abstract] [Full Text] [Related]

  • 18. Sodium-glucose cotransporter type 2 channel inhibitor: Breakthrough in the treatment of neutropenia in patients with glycogen storage disease type 1b?
    Kaczor M, Greczan M, Kierus K, Ehmke Vel Emczyńska-Seliga E, Ciara E, Piątosa B, Rokicki D, Książyk J, Wesół-Kucharska D.
    JIMD Rep; 2022 May 12; 63(3):199-206. PubMed ID: 35433171
    [Abstract] [Full Text] [Related]

  • 19. Current understanding on pathogenesis and effective treatment of glycogen storage disease type Ib with empagliflozin: new insights coming from diabetes for its potential implications in other metabolic disorders.
    Maiorana A, Tagliaferri F, Dionisi-Vici C.
    Front Endocrinol (Lausanne); 2023 May 12; 14():1145111. PubMed ID: 37152929
    [Abstract] [Full Text] [Related]

  • 20. Favorable outcome of empagliflozin treatment in two pediatric glycogen storage disease type 1b patients.
    Hexner-Erlichman Z, Veiga-da-Cunha M, Zehavi Y, Vadasz Z, Sabag AD, Tatour S, Spiegel R.
    Front Pediatr; 2022 May 12; 10():1071464. PubMed ID: 36507137
    [Abstract] [Full Text] [Related]

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